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COMPOUNDS TARGETING PAX3::FOXO1 FUSION PROTEIN

Application US20260083724A1 Kind: A1 Mar 26, 2026

Assignee

Georgetown University

Inventors

Aykut Üren, Jeffrey A. Toretsky

Abstract

Compounds comprising a PAX3::FOXO1 fusion protein binding moiety, a bivalent linker, and an E3 ubiquitin ligase binding moiety. The compounds can be used to reduce PAX3::FOXO1 protein levels in a subject with fusion-positive rhabdomyosarcoma (FP-RMS), to ubiquitinate a PAX3::FOXO1 fusion protein in a cell, to treat FP-RMS in a subject in need thereof, and/or to treat a disease caused by the overexpression of PAX3::FOXO1 protein in a subject in need thereof.

CPC Classifications

A61K 31/4709 A61P 35/00

Filing Date

2025-09-26

Application No.

19341825